Combat Clara's Blindness
At 12 years old, Clara is already a unique individual. She was born with a rare disease, Lebers Congenital Amaurosis type 6 (LCA6). This causes her to be legally blind. She also has strabismus (mis-alignment), nystagmus (her eyes wiggle) and lacks the ability to decipher colors, details and depth perception. She reads Braille and uses a cane. Despite her challenges, she lives life to the fullest. Clara loves being the center of attention, especially if it is on a stage. She has fine tuned her ears to play the piano, flute and sing. Cheering, Marching Band, Horse Riders Club, Girl Scouts and 4-H are a few of her after school activities. Clara has never let her blindness stop her, but this gene therapy will preserve the little vision she does have and quite possibly improve it. Without this treatment, this degenerative disease will take away the vision that she does have.
LCA6 caused by mutations in RPGRIP1 is a rare disease, so there is very little commercial interest because the perceived profits are too low. It is frustrating to know that a possible treatment has been discovered but isn’t being developed because there is a lack of funding.
Funds raised will enable development of a treatment. Your donation will directly support manufacturing and safety testing for clinical trials. People like you and me are coming together to support this program because there is no commercial interest. The future of this program depends on private support. In total the project needs $2 million to develop the drug, perform clinical trials and begin to treat patients by 2025.
I know there are many fundraisers out there and I truly appreciate your consideration. Every bit will help and together will make a difference for people who have this rare disease.